Cogent enveloped its application to the FDA

Announcement

Cogent Biosciences, Inc., a leading biotechnology company dedicated to establishing accurate therapies for genetically defined diseases, has now submitted its promising New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the company’s best-in-class bezuclastinib in NonAdvanced Systemic Mastocytosis (NonAdvSM). This submission is related to positive clinical data from Cogent’s SUMMIT pivotal trial. The submission follows the footprint of the Breakthrough Therapy Designation for bezuclastinib in SSM and NonAdvSM patients who have received it before avapritinib.

Cogent’s Chief Executive Officer and President, Andrew Robbins, said, “This application is the first of the three intended submissions for bezuclastinib related to positive clinical data from its three completed pivotal trials in 2025 for GIST and systemic mastocytosis patients. Grounding on the exceptional results from the SUMMIT trial, this registration takes us one step closer to serving a fundamental disease-modifying therapy to NonAdvSM patients. Further, we are thankful to the families, clinicians, patients, our team and collaborators for being supportive throughout the process.”

Bezuclastinib’s potential

Bezuclastinib illustrated a crystal clear clinical advantage throughout each symptom domain, involving noteworthy improvements in 11 patients who showed symptoms and the extreme symptom at the foundation level. The mitigation in the objective measures of condition involving serum tryptase associated with the improvements in the symptom severity marks the effectiveness and trust of the NonAdvSM patients. In 2025, currently at the ASH annual meeting, the SUMMIT trial of bezuclastinib in NonAdvSM patients gained a statistically meaningful value in all major primary and secondary endpoints.

The advanced data from the SUMMIT trial, over the long 48 weeks of the trial, certified its potential value and continued to intensify the symptomatic improvement to date. The trial contributes to the ability for a sustainable clinical benefit with extensional timeline of therapy. Throughout the SUMMIT trial, bezuclastinib illustrated a beneficial tolerability and safety profile that expands the strength and ability for chronic use in NonAdvSM patients.

In October 2025, the bezuclastinib was awarded a Breakthrough Therapy Designation by the FDA. This designation mirrors the agency’s recognition of its capability to identify a noteworthy unmet medical need. This NDA submission in AdvSM and GIST is on the perfect track to land in the first half of 2026, based on the power of convincing of the clinical results from the APEX and PEAK clinical trials, as noted in Q4 2025.